ISSUE: Early treatment represents an opportunity for prevention of the long-term complications and economic burden associated with many chronic progressive diseases. Estimating the full societal value of early preventive treatment is essential to ensuring patient access and incentivizing innovation. Treatments with high upfront costs, large target populations, and/or significant lags before benefit accrual also may require innovative payment models to ensure affordability. Recent commentaries on value assessment and reimbursement challenges in conditions such as Alzheimer’s disease and for one-time curative gene therapies suggest that traditional economic evaluation approaches and payment models may not be up to the task. In this issue panel, we propose that these challenges can be generalized to other early preventive treatments for chronic progressive diseases and debate the appropriateness of traditional value assessment and reimbursement approaches. OVERVIEW: Dr. Herring will introduce the topic and conduct an audience poll on initial perceptions (9 minutes). Dr. Cole will consider the payer’s perspective and reflect on methodologies employed by the National Institute for Health and Care Excellence in the United Kingdom, their appropriateness, and what evolution in payment models may be required to support and incentivize innovation (12 minutes). Dr. Phelps will advocate for the use of the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) approach, explicitly addressing applications of GRACE when severity increases with time and presenting on the incorporation of caregiver impacts in GRACE (12 minutes). Dr. Jiao will present and review emerging methods for incorporating broader, non-health benefits into value assessment, focusing on potential productivity, financial risk protection, and equity benefits, illustrated by recent evaluations of gene therapy for sickle cell disease (12 minutes). We will conclude with a discussion among the panelists and the audience, including a final audience poll (15 minutes). This issue panel may benefit payers, economic modelers, and other stakeholders involved in value assessment and reimbursement decisions.
